(S)-(+)-4-[7-(2,2-Dimethyl-1-oxopro- poxy)-4-methyl-2-[4-[2-(1-piperidinyl)- ethoxy]phenyl]-2H-1-benzopyran-3-yl]- phenyl 2,2-Dimethylpropanoate (EM-800):  A Highly Potent, Specific, and Orally Active Nonsteroidal Antiestrogen

Journal of Medicinal Chemistry
1997.0

Abstract

Breast cancer is the most frequent cancer and the second cause of cancer death in women in North America, with available therapies showing limitations. Tamoxifen, a widely used antiestrogen, has mixed agonist-antagonist activities, and steroidal antiestrogens like ICI 164,384 have limited oral bioavailability. We synthesized a new nonsteroidal antiestrogen, (S)-(+)-4-[7-(2,2-Dimethyl-1-oxopropoxy)-4-methyl-2-[4-[2-(1-piperidinyl) ethoxy]phenyl]-2H-1-benzopyran-3-yl] phenyl 2,2-Dimethylpropanoate (EM-800, (S)-1), via resolution of racemic chromene 6 and prodrug derivatization of the active enantiomer (S)-6 (EM-652). We compared the biological properties of enantiomers (R)-1/(R)-6 and (S)-1/(S)-6. Results showed (S)-6/(S)-1 had much higher estrogen receptor affinity (e.g., (S)-6 had Ki 0.047 nM in human breast cancer cytosol, relative binding affinity (RBA) 291 vs estradiol's 100) and potency in inhibiting estradiol-stimulated proliferation of T-47D and ZR-75-1 human breast cancer cells (e.g., (S)-1 had IC50 0.14 nM vs (R)-1's 36.2 nM in T-47D cells, 260-fold higher potency). In vivo, (S)-1 was at least 30 times more potent than (R)-1 in inhibiting estrone-stimulated uterine weight in ovariectomized mice, and acted as a pure estrogen antagonist in mammary gland, endometrium, and hypothalamopituitary feedback without agonist activity. Additionally, EM-800 increased bone mineral density and decreased serum cholesterol and triglycerides in rats. EM-800 [(S)-1] is the most potent antiestrogen described so far, with high specificity and oral activity. The availability of such a potent and specific antiestrogen could lead to significant improvement in the treatment of estrogen-sensitive breast and uterine cancer as well as nonmalignant estrogen-sensitive diseases.

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